Oral Presentation 2015 Annual Meeting of the Australasian Society for Dermatology Research

Characterising the severity and responsiveness of a new outcome measure for epidermolysis bullosa – The Epidermolysis Bullosa Disease Activity and Scarring Index  (#26)

Swaranjali V Jain 1 2 , Adam G Harris 2 , John S Su 3 , David Orchard 3 , Lachlan J Warren 4 , Hamish McManus 5 , Dedee F Murrell 1 2
  1. UNSW, Kensington, NSW, Australia
  2. Dermatology, St George Hospital, Sydney
  3. Dermatology, The Royal Children's Hospital, Melbourne
  4. Dermatology, Women's and Children's Hospital, Adelaide, South Australia, Australia
  5. Kirby Institute, University of New South Wales, Sydney

Introduction: The development of novel therapies for epidermolysis bullosa (EB) necessitates validated outcome measures that can capture disease severity. The Epidermolysis Bullosa Disease Activity and Scarring Index (EBDASI) was developed to measure disease activity and damage in clinical trials.1 The aim of this study was to assess the responsiveness of the EBDASI by characterising disease severity and the clinical response of epidermolysis bullosa over time.

Methods: 48 patients with EB attending routine outpatient clinics at three institutions were prospectively evaluated. Patients at each visit were evaluated by the principal investigator at each site using the EBDASI, a physician assessment of disease severity (mild, moderate, severe) and a physician assessment of change compared to the previous visit. Site-specific analysis determined EBDASI score ranges for patients in each severity category and correlated scores with an EB-specific quality-of-life measure. The minimal clinically important difference of the EBDASI activity score was calculated in the responsiveness analysis. Receiver operating characteristic (ROC) curves were used in all analyses to determine score cut-offs.

Results: An EBDASI score cut-off of 42 differentiated mild from moderate disease and a cut-off of 107 differentiated severe from moderate disease. Reduction in EBDASI activity scores of greater than 7 and increase in scores of greater than 3 corresponded with clinical improvement and deterioration, respectively. All cut-offs correctly classified approximately 80% of patients.

Conclusions: The EBDASI can be used to categorise patients according to severity and clinical response and is promising as an outcome measure for future clinical trials.

  1. Loh, C. C. H., Kim, J., Su, J. C., Daniel, B. S., Venugopal, S. S., Rhodes, L. M., et al. (2014). Development, reliability, and validity of a novel Epidermolysis Bullosa Disease Activity and Scarring Index (EBDASI). Journal of the American Academy of Dermatology, 70(1), 89-97.